Healing Parkinson's patients with their own stem cells

Point Loma  A groundbreaking stem cell treatment for Parkinson’s disease is getting close to moving from lab research in La Jolla to therapy for patients. The research, funded by the patients and their supporters, could also pioneer a new model for moving medical advances from the lab into the clinic.

Eight Parkinson’s patients have allied with scientists from The Scripps Research Institute and medical professionals from Scripps Clinic for the project, which involves creating new brain cells from other cells in their own bodies. Because of the unusual, personalized nature of the research, the patients are participating with scientists and doctors as equals, meeting regularly to review the progress.

The ambitious goal is to relieve the movement difficulties Parkinson’s causes by replacing the brain cells the disease destroys. In theory, it would restore near-normal movement for a decade or more, and the procedure could be repeated as needed.

Research is far enough along that scientists and health care professionals in the project are talking to regulators about beginning clinical trials, perhaps as soon as next year.

The replacement brain cells are now being grown in a lab at The Scripps Research Institute. Patches of skin the diameter of a pencil eraser were removed from the patients’ arms and turned into a new kind of stem cell that acts like embryonic stem cells. Called induced pluripotent stem cells, they were discovered in 2006, a feat honored by a Nobel Prize last year.

These IPS cells can become nearly any kind of cell in the body, but they don’t raise the ethical concerns of using embryonic stem cells. Another potential advantage of IPS cells over embryonic stem cells is that they should be less prone to rejection by the patients’ immune systems, because the transplanted cells come from the individuals themselves.

IPS cells are not derived from human embryos

The personalized nature of the therapy, however, has a significant downside. It takes months to grow the needed cells for each patient. That’s unappealing to drug companies, who want treatments that can be mass-produced. And because the technology is so new, getting government grants hasn’t been possible, say scientists on the project.

So the eight patients and their supporters are raising the money to finance the research themselves, under the name of Summit4StemCell, operating under the nonprofit aegis of the Parkinson’s Association of San Diego.

So far, the patients have raised $453,000. The project’s next stage is expected to cost more than $5 million.

Summit4StemCell continually raises money from events, the most prominent to date being a climb up Mount Kilimanjaro in Africa, with patients who are physically able. This October, they plan to climb to base camp at Mount Everest.

Jeanne Loring, director of the Center for Regenerative Medicine at The Scripps Research Institute, and Andres Bratt-Leal, a member of Loring's lab who works on the stem cell project for eight Parkinson's disease patients.
— HOWARD LIPIN/U-T San Diego/ZUMA PRESS, U-T San Diego

Jeanne Loring, director of the Center for Regenerative Medicine at The Scripps Research Institute, and Andres Bratt-Leal, a member of Loring's lab who works on the stem cell project for eight Parkinson's disease patients.
/ HOWARD LIPIN/U-T San Diego/ZUMA PRESS, U-T San Diego

The project’s research is led by Jeanne Loring, head of the Center for Regenerative Medicine at The Scripps Research Institute. Neurologist Melissa Houser, clinical director of the Parkinson’s Disease and Movement Disorder Center at Scripps Clinic, leads the medical side. Nurse practitioner Sherrie Gould, who works with Houser, co-ordinates with patients. Gould is Summit4StemCell’s sparkplug: The climb up Mount Kilimanjaro was her idea.